Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles
Abstract Inefficient targeting of muscle stem cells (MuSCs), also called satellite cells, represents a major bottleneck of current therapeutic strategies for muscular dystrophies, as it precludes the possibility of promoting compensatory regeneration. Here we describe a muscle-targeting delivery pla...
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| Format: | Article |
| Language: | English |
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Nature Portfolio
2025-01-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-024-55223-9 |
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| author | Francesco Millozzi Paula Milán-Rois Arghya Sett Giovanni Delli Carpini Marco De Bardi Miguel Gisbert-Garzarán Martina Sandonà Ciro Rodríguez-Díaz Mario Martínez-Mingo Irene Pardo Federica Esposito Maria Teresa Viscomi Marina Bouché Ornella Parolini Valentina Saccone Jean-Jacques Toulmé Álvaro Somoza Daniela Palacios |
| author_facet | Francesco Millozzi Paula Milán-Rois Arghya Sett Giovanni Delli Carpini Marco De Bardi Miguel Gisbert-Garzarán Martina Sandonà Ciro Rodríguez-Díaz Mario Martínez-Mingo Irene Pardo Federica Esposito Maria Teresa Viscomi Marina Bouché Ornella Parolini Valentina Saccone Jean-Jacques Toulmé Álvaro Somoza Daniela Palacios |
| author_sort | Francesco Millozzi |
| collection | DOAJ |
| description | Abstract Inefficient targeting of muscle stem cells (MuSCs), also called satellite cells, represents a major bottleneck of current therapeutic strategies for muscular dystrophies, as it precludes the possibility of promoting compensatory regeneration. Here we describe a muscle-targeting delivery platform, based on gold nanoparticles, that enables the release of therapeutic oligonucleotides into MuSCs. We demonstrate that AuNPs conjugation to an aptamer against α7/β1 integrin dimers directs either local or systemic delivery of microRNA-206 to MuSCs, thereby promoting muscle regeneration and improving muscle functionality, in a mouse model of Duchenne Muscular Dystrophy. We show here that this platform is biocompatible, non-toxic, and non-immunogenic, and it can be easily adapted for the release of a wide range of therapeutic oligonucleotides into diseased muscles. |
| format | Article |
| id | doaj-art-6c47b54fe28f4bbba5b46a3839868074 |
| institution | Kabale University |
| issn | 2041-1723 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Nature Communications |
| spelling | doaj-art-6c47b54fe28f4bbba5b46a38398680742025-01-12T12:30:19ZengNature PortfolioNature Communications2041-17232025-01-0116111910.1038/s41467-024-55223-9Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic musclesFrancesco Millozzi0Paula Milán-Rois1Arghya Sett2Giovanni Delli Carpini3Marco De Bardi4Miguel Gisbert-Garzarán5Martina Sandonà6Ciro Rodríguez-Díaz7Mario Martínez-Mingo8Irene Pardo9Federica Esposito10Maria Teresa Viscomi11Marina Bouché12Ornella Parolini13Valentina Saccone14Jean-Jacques Toulmé15Álvaro Somoza16Daniela Palacios17Department of Life Science and Public Health, Università Cattolica del Sacro CuoreIMDEA NanocienciaBordeaux University, Inserm U1212Department of Life Science and Public Health, Università Cattolica del Sacro CuoreFondazione Santa Lucia IRCCSIMDEA NanocienciaDepartment of Life Science and Public Health, Università Cattolica del Sacro CuoreIMDEA NanocienciaIMDEA NanocienciaIMDEA NanocienciaDepartment of Anatomical, Histological, Forensic Medicine and Orthopaedic Sciences, Section of Histology and Embryology, Sapienza University of RomeDepartment of Life Science and Public Health, Università Cattolica del Sacro CuoreDepartment of Anatomical, Histological, Forensic Medicine and Orthopaedic Sciences, Section of Histology and Embryology, Sapienza University of RomeDepartment of Life Science and Public Health, Università Cattolica del Sacro CuoreDepartment of Life Science and Public Health, Università Cattolica del Sacro CuoreBordeaux University, Inserm U1212IMDEA NanocienciaDepartment of Life Science and Public Health, Università Cattolica del Sacro CuoreAbstract Inefficient targeting of muscle stem cells (MuSCs), also called satellite cells, represents a major bottleneck of current therapeutic strategies for muscular dystrophies, as it precludes the possibility of promoting compensatory regeneration. Here we describe a muscle-targeting delivery platform, based on gold nanoparticles, that enables the release of therapeutic oligonucleotides into MuSCs. We demonstrate that AuNPs conjugation to an aptamer against α7/β1 integrin dimers directs either local or systemic delivery of microRNA-206 to MuSCs, thereby promoting muscle regeneration and improving muscle functionality, in a mouse model of Duchenne Muscular Dystrophy. We show here that this platform is biocompatible, non-toxic, and non-immunogenic, and it can be easily adapted for the release of a wide range of therapeutic oligonucleotides into diseased muscles.https://doi.org/10.1038/s41467-024-55223-9 |
| spellingShingle | Francesco Millozzi Paula Milán-Rois Arghya Sett Giovanni Delli Carpini Marco De Bardi Miguel Gisbert-Garzarán Martina Sandonà Ciro Rodríguez-Díaz Mario Martínez-Mingo Irene Pardo Federica Esposito Maria Teresa Viscomi Marina Bouché Ornella Parolini Valentina Saccone Jean-Jacques Toulmé Álvaro Somoza Daniela Palacios Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles Nature Communications |
| title | Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles |
| title_full | Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles |
| title_fullStr | Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles |
| title_full_unstemmed | Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles |
| title_short | Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles |
| title_sort | aptamer conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles |
| url | https://doi.org/10.1038/s41467-024-55223-9 |
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